Pharmaceutical Technology Editors

Moderna announced on Oct. 14, 2021, that FDA’s Vaccines and Related Biological Products Advisory Committee (VRBPAC) unanimously recommended that FDA grant an Emergency Use Authorization (EUA) for a booster dose of the Moderna COVID-19 vaccine (mRNA-1273). In a 19–0 vote, the VRBPAC recommended a 50 ug booster shot should be made available to three populations:

People aged 18 to 64 who are at high risk for severe COVID-19

People aged 18 to 64 whose exposure to COVID-19 puts them at risk for COVID-19 complications or severe illness.

Additionally, this booster dose is to be administered only after at least six months have passed since their original vaccination series.

A recent Phase II study of mRNA-1273 was conducted wherein 344 interested participants were given a 50-ug booster dose six to eight months following their second dose. According to the study, neutralizing antibody titers, particularly against variants of concern, had waned prior to this study. However, following a booster dose, neutralizing titers were raised significantly to the Phase III benchmark level.

“We thank the committee for their review and for their positive recommendation in support of [EUA]. This positive recommendation is supported by data on the 50-µg booster dose of our COVID-19 vaccine, which shows robust antibody responses against the original virus, but also against the Delta variant,” said Stéphane Bancel, CEO of Moderna, in a company press release. “We remain committed to staying ahead of the virus and following the evolving epidemiology of SARS-CoV-2. We look forward to making our booster available to people in the U[nited] S[tates] to help protect themselves against this ongoing public health emergency.”

This recommendation is non-binding but will be taken into consideration as it concerns an EUA. The Centers for Disease Control and Prevention’s Advisory Committee on Immunization Practices will also meet to discuss a recommendation on a booster shot.

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The council voted 19–0 to recommend that FDA authorize booster doses of the Moderna COVID-19 vaccine for certain populations.

FDA Advisory Council Unanimously Supports Moderna COVID-19 Booster EUA

Poseida Therapeutics, a clinical-stage biopharmaceutical company specializing in cell and gene therapeutics, announced that it has entered into a research collaboration and exclusive license agreement with Takeda Pharmaceutical Company in an Oct. 12, 2021, press release. In this collaboration, Takeda will utilize Poseida's nanoparticle delivery technology and other proprietary genetic engineering platforms for the research and development of up to eight gene therapies.

This collaboration will focus on developing non-viral 

 gene therapy programs, including Poseida's Hemophilia A program. Poseida will lead research activities up to candidate selection, after which Takeda will assume responsibility for further development and commercialization.

The parties will collaborate to initially develop up to six 

gene therapy programs under the terms of the agreement, utilizing Poseida's technology platforms—including piggyBac, Cas-CLOVER, and biodegradable nanoparticle technology as well as other technologies. In addition, Takeda can add two additional programs to the collaboration and would then provide funding for all collaboration program R&D costs. Poseida will receive an upfront payment of $45 million and preclinical milestones and is eligible to receive future clinical development, regulatory, and commercial milestone payments.

"Our technologies offer highly efficient gene delivery, fully integrated non-viral genome insertion, and ultra-precise site-specific gene editing,” said Eric Ostertag, CEO of Poseida, in a company press release. “Together with Takeda, we look forward to developing potential cures for a number of genetic diseases with high unmet need."

"Poseida's differentiated platform technologies show great promise in developing non-viral 

 gene therapies using their novel genetic engineering and delivery technologies that complement our existing collaborations," said Madhu Natarajan, head of Takeda’s rare diseases drug discovery unit, in the press release.

In this collaboration, Takeda will utilize Poseida's biodegradable DNA and RNA nanoparticle delivery technology and other proprietary genetic engineering platforms for the research and development of gene therapies.

Poseida Therapeutics and Takeda Partner to Develop Non-Viral In-Vivo Gene Therapy Programs

MilliporeSigma, the United States and Canadian life science business of Merck KGaA, Darmstadt, Germany, announced on Oct. 12, 2021, the opening of its second Carlsbad, Calif. facility, which expands its footprint as a contract development and manufacturing organization (CDMO). The $110 million, 140,000-square-footfacility will more than double the life science business sector’s existing capacity to support large-scale commercial and industrial manufacturing for viral gene therapy.

This is MilliporeSigma’s second Carlsbad, Calif. facility, both of which will focus on cell and gene therapy—specifically viral vector-based therapies. There are 30 cleanroom suites between the two facilities that support small- to large-scale clinical and commercial production. In addition, MilliporeSigma is one of the first CDMOs granted FDA and European Medicines Agency approval for viral vector manufacturing, according to the company press release.

“Today, most gene therapies target rare diseases, but there is an urgent need to efficiently manufacture these treatments to reach larger patient populations,” said Matthias Heinzel, CEO, Life Science, Merck, in the press release. “As a leader in viral vector manufacturing, this increase in capacity and scale is the next step in enabling our customers to bring new curative treatments to market.”

MilliporeSigma announced the opening of its second Carlsbad, California-based facility, which doubles production capacity to support commercial and industrial manufacturing of viral vectors. 

MilliporeSigma Completes $110-Million Contract Development Manufacturing Facility for Gene Therapy

Aceto, a provider of specialty materials, announced its acquisition of A&C Bio Buffer, a good manufacturing practices (GMP) manufacturer of custom buffer and chemical blend products used in the manufacturing of biopharmaceutical drug products, in an Oct. 12, 2021, press release. At A&C Bio Buffer’s ISO 9001:2015-certified facility in Limerick, Ireland, the company manufactures more than 50 products, including biological buffers, process solutions, cleaning solutions, and water for injection.

“A&C Bio Buffer is a natural add-on to our acquisition of A&C and aligns with our commitments to the biopharmaceutical space,” said Gilles Cottier, CEO of Aceto, in the press release. “It is a nimble, highly customer-centric organization serving some of the top biopharmaceutical manufacturers in Europe. Its rapid growth reflects a reputation for service and quality, which recently led to its key role in the raw material supply chain for COVID-19 vaccines. A&C Bio Buffer enhances our growing European manufacturing footprint and commercial presence. We believe the shared lineage reflected in A&C Bio Buffer’s skills, culture and customer focus will further support our evolution as a leading supplier of critical raw materials in the life sciences sector.”

Earlier this year, Aceto also acquired A&C, a global manufacturer of GMP specialty excipients and custom process solutions. According to Aceto’s press release, the company will continue to support A&C BioBuffer’s manufacturing facilities and offices in Ireland. However, financial terms were not disclosed.

Aceto will acquire A&C Bio Buffer, including the company’s custom buffer and chemical blend portfolio.

Aceto Acquires A&C Bio Buffer, Expanding GMP Product Lines in Biopharma

PharmaLex Group announced the formal launch on Oct. 12, 2021, of the Biopharma Excellence brand, a major new service line that fuses the scientific, regulatory, and commercialization expertise of PharmaLex, ERA Consulting, and Biopharma Excellence. Christian K. Schneider, the former scientific lead of the Medicines and Healthcare products Regulatory Agency, will head the new unit as chief medical officer.

This new service line combines over 35 years of empirical experience and respected regulator relationships and has a global team of over 70 scientific, regulatory, and commercial professionals that can offer strategic product development and proactive regulatory services that are aimed specifically at developers of advanced therapy medicinal products (ATMPs).

The launch of Biopharma Excellence coincides with the maturation of the biopharma industry, in which the development of ATMPs has accelerated, giving rise to commercial opportunity for innovation in fields such as cell and gene therapies, monoclonal antibodies, vaccines, and biosimilars.

Biopharma Excellence is intended to serve biopharmaceutical companies ranging from small startups to divisions of large, established life sciences organizations. Its multi-disciplinary solutions are geared toward commercial as well as scientific and regulatory success.

“Gene and cell therapies have reached ‘adolescence’ now,” said Schneider, who was previously chair of the European Medicines Agency’s Committee for Advanced Therapies, in a company press release. “Ten years ago, there wasn’t a single treatment authorized, but now we see numerous successes emerging from clinical trials, or treatments already authorized, from CAR [chimeric antigen receptor] T-cell therapy to gene therapies designed to correct rare, hereditary diseases.”

“This, in turn, is opening the door for innovative biopharmaceutical developers. But because their ambitions invariably involve difficult clinical indications, their route to market is far from straightforward—which is where Biopharma Excellence comes in. Our combined breadth of knowledge, and the ability to apply this strategic, multi-disciplinary scientific, regulatory and commercial expertise to biopharma specifically, makes for a very powerful proposition,” Schneider added. “I’d like to see Biopharma Excellence become a clear leader in biotech: in actively enabling the advancement of cutting-edge medicines. We’re very well placed for this, and I hope my 15 years at the front end of Regulatory Science will further help to ensure that our clients strike the right balance between meeting Agency scientific requirements and creating a compelling proposition for investors and payers.”

Pharmalex has launched the Biopharma Excellence brand, a new service line that combines the expertise of PharmaLex, ERA Consulting, and Biopharma Excellence.

Pharmalex Launches Major New Service Line, Biopharma Excellence

SGS announced on Oct. 12, 2021, that it is investing in a new extractables and leachables (E&L) testing laboratory in Navi Mumbai, India. The lab is set to open by the end of 2021 and will provide testing services for verifying product safety for pharmaceutical, biopharmaceutical, and medical device companies in Southeast Asia.

The new lab will be approximately 3000 ft.2 and will offer a broad spectrum of the latest E&L testing solutions, including:

gas chromatography–mass spectrometry (GC–MS) and headspace–GC/MS for the analysis of semi-volatile and volatile components

liquid chromatography with tandem mass spectrometry (LC–MSMS) for the quantitative analysis of trace leachable components

high resolution LC–MSMS to generate data that supports identification of unknown components

and inductively coupled plasma–MS to quantitate screening and identification of inorganics.

This laboratory in India will complement SGS's existing testing network. The company has three other E&L centers of excellence located in the United States, Germany, and China.

SGS is investing in a new E&L lab in Navi Mumbai, India, that will provide testing services.